The Food and Drug Administration (FDA) has approved Ocaliva (obeticholic acid; Intercept) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid in adults with inadequate response to ursodeoxycholic acid, or as a single therapy in adults unable to tolerate ursodeoxycholic acid. 

Ocaliva is an oral farnesoid X receptor agonist, a receptor found on the nucleus of cells in the liver and intestine. The farnesoid X receptor is involved in the bile acid metabolic pathways. Ocaliva increases bile flow from the liver and suppresses bile acid production in the liver, thus decreasing the liver’s exposure to toxic levels of bile acids. 

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The FDA’s accelerated approval was based on a reduction in alkaline phosphatase (ALP) levels, as a surrogate endpoint which could be relied upon to be reasonably likely to predict clinical benefit, including an improvement in transplant-free survival. The safety and efficacy of Ocaliva were evaluated in POISE, a 12-month, double-blind, randomized-controlled trial (n=216) where the proportion of patients achieving ALP reductions was higher among Ocaliva-treated patients vs. placebo-treated patients after 12 months (46% vs. 10%; P<0.0001). 

Ocaliva should not be used in patients with complete biliary obstruction. A confirmatory trial is currently ongoing; an improvement in survival, progression to cirrhosis, or other disease-related symptoms in patients being treated with Ocaliva has not yet been established. 

Ocaliva was granted fast track designation and an orphan drug designation by the FDA. It is available as 5mg and 10mg strength tablets in 30-count bottles. Ocaliva is expected to launch within 7–10 days and will be distributed through a specialty pharmacy network.

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